CA3062590A1 - Antisense oligonucleotides for modulating htra1 expression - Google Patents
Antisense oligonucleotides for modulating htra1 expression Download PDFInfo
- Publication number
- CA3062590A1 CA3062590A1 CA3062590A CA3062590A CA3062590A1 CA 3062590 A1 CA3062590 A1 CA 3062590A1 CA 3062590 A CA3062590 A CA 3062590A CA 3062590 A CA3062590 A CA 3062590A CA 3062590 A1 CA3062590 A1 CA 3062590A1
- Authority
- CA
- Canada
- Prior art keywords
- oligonucleotide
- nucleosides
- htra1
- region
- seq
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Abandoned
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Classifications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
- C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
- C12Y304/21—Serine endopeptidases (3.4.21)
- C12Y304/21108—HtrA2 peptidase (3.4.21.108)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
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- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/33—Chemical structure of the base
- C12N2310/334—Modified C
- C12N2310/3341—5-Methylcytosine
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/341—Gapmers, i.e. of the type ===---===
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
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| Application Number | Priority Date | Filing Date | Title |
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| EP17173964.2 | 2017-06-01 | ||
| EP17173964 | 2017-06-01 | ||
| EP17209407 | 2017-12-21 | ||
| EP17209535 | 2017-12-21 | ||
| EP17209535.8 | 2017-12-21 | ||
| EP17209407.0 | 2017-12-21 | ||
| PCT/EP2018/064221 WO2018220034A1 (en) | 2017-06-01 | 2018-05-30 | Antisense oligonucleotides for modulating htra1 expression |
Publications (1)
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| CA3062590A1 true CA3062590A1 (en) | 2018-12-06 |
Family
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| CA3062590A Abandoned CA3062590A1 (en) | 2017-06-01 | 2018-05-30 | Antisense oligonucleotides for modulating htra1 expression |
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| Country | Link |
|---|---|
| US (3) | US20190055564A1 (ko) |
| EP (1) | EP3630976A1 (ko) |
| JP (1) | JP7169995B2 (ko) |
| KR (1) | KR20200015608A (ko) |
| CN (1) | CN110691849A (ko) |
| AU (1) | AU2018277219A1 (ko) |
| BR (1) | BR112019025290A2 (ko) |
| CA (1) | CA3062590A1 (ko) |
| CL (1) | CL2019003337A1 (ko) |
| CO (1) | CO2019013317A2 (ko) |
| CR (1) | CR20190543A (ko) |
| IL (1) | IL271039A (ko) |
| MA (1) | MA49278A (ko) |
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| PE (1) | PE20191845A1 (ko) |
| PH (1) | PH12019502636A1 (ko) |
| RU (1) | RU2019143004A (ko) |
| TW (1) | TW201907008A (ko) |
| WO (1) | WO2018220034A1 (ko) |
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| CR20180606A (es) | 2016-07-01 | 2019-02-14 | Hoffmann La Roche | Oligonucleótidos antisentido para modular la expresión de htra1 |
| JP7317029B2 (ja) | 2018-02-12 | 2023-07-28 | アイオーニス ファーマシューティカルズ， インコーポレーテッド | 修飾化合物及びその使用 |
| WO2022092326A1 (ja) * | 2020-10-30 | 2022-05-05 | 英彰 原 | 眼組織線維化の阻害に使用するためのｇｄｆ１５調節物質 |
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| JP3756313B2 (ja) | 1997-03-07 | 2006-03-15 | 武 今西 | 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体 |
| IL135000A0 (en) | 1997-09-12 | 2001-05-20 | Exiqon As | Bi- and tri-cyclic nucleoside, nucleotide and oligonucleotide analogues |
| AU5290499A (en) * | 1998-08-03 | 2000-02-28 | Novartis Ag | Human htra serine protease |
| TR200604211T1 (tr) | 1999-02-12 | 2007-02-21 | Daiichi Sankyo Company Limiteddaiichi Sankyo Company Limited | Yeni nükleosid ve oligonükleotid analoglarıYeni nükleosid ve oligonükleotid analogları |
| US7053207B2 (en) | 1999-05-04 | 2006-05-30 | Exiqon A/S | L-ribo-LNA analogues |
| US6617442B1 (en) | 1999-09-30 | 2003-09-09 | Isis Pharmaceuticals, Inc. | Human Rnase H1 and oligonucleotide compositions thereof |
| DK3222722T3 (da) | 2002-11-18 | 2019-06-17 | Roche Innovation Ct Copenhagen As | Antisense-design |
| EP1888083B1 (en) * | 2005-05-24 | 2011-12-28 | Isis Pharmaceuticals, Inc. | Compositions and their uses directed to lmw-ptpase |
| WO2007031091A2 (en) | 2005-09-15 | 2007-03-22 | Santaris Pharma A/S | Rna antagonist compounds for the modulation of p21 ras expression |
| PL1984381T3 (pl) | 2006-01-27 | 2011-03-31 | Isis Pharmaceuticals Inc | Zmodyfikowane w pozycji 6 analogi bicykliczne kwasów nukleinowych |
| US7666854B2 (en) | 2006-05-11 | 2010-02-23 | Isis Pharmaceuticals, Inc. | Bis-modified bicyclic nucleic acid analogs |
| JP5441688B2 (ja) | 2006-05-11 | 2014-03-12 | アイシス ファーマシューティカルズ， インコーポレーテッド | ５’修飾二環式核酸類似体 |
| WO2008013893A2 (en) | 2006-07-26 | 2008-01-31 | Yale University | Diagnosis and treatment of age related macular degeneration |
| WO2008067040A2 (en) * | 2006-10-06 | 2008-06-05 | University Of Utah Research Foundation | Method of detecting ocular diseases and pathologic conditions and treatment of same |
| WO2008150729A2 (en) | 2007-05-30 | 2008-12-11 | Isis Pharmaceuticals, Inc. | N-substituted-aminomethylene bridged bicyclic nucleic acid analogs |
| EP2173760B2 (en) | 2007-06-08 | 2015-11-04 | Isis Pharmaceuticals, Inc. | Carbocyclic bicyclic nucleic acid analogs |
| TW200911290A (en) * | 2007-07-02 | 2009-03-16 | Alcon Res Ltd | RNAI-mediated inhibition of HTRA1 for treatment of macular degeneration |
| EP2176280B2 (en) | 2007-07-05 | 2015-06-24 | Isis Pharmaceuticals, Inc. | 6-disubstituted bicyclic nucleic acid analogs |
| EP2217720A4 (en) * | 2007-11-01 | 2010-12-08 | Univ Iowa Res Found | RNA LOCUS ANALYSIS TO ASSESS THE STUNNING FOR AMD AND MPGNII |
| US8546556B2 (en) | 2007-11-21 | 2013-10-01 | Isis Pharmaceuticals, Inc | Carbocyclic alpha-L-bicyclic nucleic acid analogs |
| WO2010036698A1 (en) | 2008-09-24 | 2010-04-01 | Isis Pharmaceuticals, Inc. | Substituted alpha-l-bicyclic nucleosides |
| EP2462153B1 (en) | 2009-08-06 | 2015-07-29 | Isis Pharmaceuticals, Inc. | Bicyclic cyclohexose nucleic acid analogs |
| US8846637B2 (en) | 2010-06-08 | 2014-09-30 | Isis Pharmaceuticals, Inc. | Substituted 2′-amino and 2′-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom |
| FR2965278B1 (fr) * | 2010-09-23 | 2014-10-10 | Univ Caen Basse Normandie | Procede d'obtention in vitro ou ex vivo de chondrocytes et leurs utilisations |
| CN104136451A (zh) | 2011-09-07 | 2014-11-05 | 玛瑞纳生物技术有限公司 | 具有构象限制的单体的核酸化合物的合成和用途 |
| WO2013154798A1 (en) | 2012-04-09 | 2013-10-17 | Isis Pharmaceuticals, Inc. | Tricyclic nucleic acid analogs |
| US20150141320A1 (en) * | 2012-05-16 | 2015-05-21 | Rana Therapeutics, Inc. | Compositions and methods for modulating gene expression |
| AU2013346767B2 (en) | 2012-11-15 | 2019-04-11 | Roche Innovation Center Copenhagen A/S | Oligonucleotide conjugates |
| US20190284621A1 (en) * | 2016-11-11 | 2019-09-19 | Roche Innovation Center Copenhagen A/S | Therapeutic oligonucleotides capture and detection |
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| BR112019025290A2 (pt) | 2020-06-23 |
| WO2018220034A1 (en) | 2018-12-06 |
| US20200157546A1 (en) | 2020-05-21 |
| TW201907008A (zh) | 2019-02-16 |
| EP3630976A1 (en) | 2020-04-08 |
| US20190055564A1 (en) | 2019-02-21 |
| MX2019014187A (es) | 2020-01-27 |
| CN110691849A (zh) | 2020-01-14 |
| AU2018277219A1 (en) | 2019-12-19 |
| PE20191845A1 (es) | 2019-12-31 |
| CL2019003337A1 (es) | 2020-05-15 |
| US20220042022A1 (en) | 2022-02-10 |
| CO2019013317A2 (es) | 2020-01-17 |
| PH12019502636A1 (en) | 2020-06-08 |
| JP7169995B2 (ja) | 2022-11-11 |
| JP2020521491A (ja) | 2020-07-27 |
| IL271039A (en) | 2020-01-30 |
| RU2019143004A (ru) | 2021-07-09 |
| MA49278A (fr) | 2020-04-08 |
| KR20200015608A (ko) | 2020-02-12 |
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